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Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)

NCT00590538 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2011-06-30

Study results available
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Summary

The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most common genetic mutation found in patients with CF is called Delta F508. Due to this mutation, there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines, pancreas and sweat glands. This lack of movement causes the clinical manifestations of the disease.

Although Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein is a naturally occurring substance that is found in food products such as soy and tofu, but is also an investigational drug for this study. When used together, both drugs may be able to restore normal chloride and salt (water) movements in body organs and glands in people with CF.

We will be studying salt and water movement in the nose by a technique called nasal transepithelial potential difference (NPD).

Conditions

Interventions

DRUG

Sodium 4-Phenylbutyrate

The standard oral adult dose is 20 g/day (tablets) for 4 days.

DRUG

Genistein (Unconjugated Isoflavones 100)

Every participant will be administered a perfusion of 50 MicroM of Genistein (Unconjugated Isoflavones 100) during the modified NPD procedure.

DRUG

Placebo

The placebo is given to match the active comparator for four days.

Sponsors & Collaborators

Principal Investigators

  • Ronald Rubenstein, M.D., PhD. · Children's Hospital of Philadelphia

Study Design

Allocation
RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-02-28
Primary Completion
2008-12-31
Completion
2008-12-31

Countries

  • United States

Study Locations

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Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00590538 on ClinicalTrials.gov